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Filter by:ItaliTTP is an observational, prospective, single-arm, national, multicenter, non-pharmacological cohort study aimed at better defining and understanding the natural history, disease severity, and clinical outcomes of patients with immune-mediated thrombotic thrombocytopenic purpura (iTTP) in Italy. A minimum of 132 consecutive patients with acute iTTP (first event or relapse) will be enrolled for 3 years, with the possibility of extension, with a follow-up period of 3 years.
This is an open-label, multicenter study to evaluate the safety, tolerability, and efficacy of HMPL-523 in adult subjects with ITP.
Immune thrombotic thrombocytopenic purpura (iTTP) is caused by a severe, autoantibody-mediated deficiency of ADAMTS13 leading to an accumulation of ultra-large von Willebrand factor multimers in plasma and finally to microthrombi in blood vessels. The current standard of care of iTTP consists in the triple association of daily plasma exchange (PEX, 60 ml/kg/day), immunosuppressive agents and anti-adhesive treatment (Caplacizumab). Our group recently reported the outcome of 90 patients with iTTP treated with this triple association and when compared to historical patients, the triplet regimen prevented death, refractoriness and exacerbations. Likewise, plasma volumes were reduced by 2 to 3-fold and we could reduce the median number of PEX sessions from 13 to 6. PEX is an invasive and time-consuming procedure, associated with catheter and plasma-related complications ranging from 22% to 30%. Consequently, to alleviate the burden of care in iTTP, using a regimen without PEX would represent a major and topical goal. Attempts to treat patients with plasma infusion (PI) without PEX were previously reported and provided evidence that large volumes of PI (20-30 ml/kg/day) improved the initial outcome of iTTP. However, fluid overload occurred in most cases after 5-7 days, limiting the feasibility of this strategy. Nevertheless, the recent availability of caplacizumab opens the perspective of treating patients with plasma for a shorter period. Recently, strategies without PEX have been carried out in Jehovah's Witnesses with iTTP [5]. Impressively, improvement was rapid and comparable to those provided with a standard PEX-based treatment. Additionally, a treatment combining caplacizumab and immunosuppression only was successfully performed in six iTTP patients with severe neurologic and/or cardiac involvement. The rapid and durable improvement provides evidence that a regimen without plasma seems feasible. However, we consider that robust data are still lacking to completely remove plasmatherapy from iTTP management. Based on these statements, we wish here to address the efficacy and safety of a PEX-free regimen, combining PI only (15 ml/kg/day), corticosteroids/rituximab, and caplacizumab.
Immune thrombocytopenia Purpura (ITP) is an autoimmune condition delineated by humoral as well as cell mediated immune response against thrombocyte surface proteins GPIIb/IIIa receptors, affecting primary homeostasis leading to mucocutaneous bleeding.ITP is characterized by platelet count <100 x 109/L. The conventional line of treatment for newly diagnosed ITP is steroids but significant disadvantages have been associated with long term use and a high risk of relapse when reducing the dose. The addition of MMF to the first line treatment of ITP resulted in substantial response and a lower risk of refractory ITP with decreased financial burden and improved outcome.
This exploratory study is to investigate the efficacy, safety and tolerability of Lusutrombopag in the treatment of primary immune thrombocytopenia in Chinese patients who have failed first-line therapy
1. Assesement of response to Thrombopoietin receptor agonists (TPO-RAs) as treatment in Idiopathic thrombocytopenia purpura patients in Assiut University hospital. 2. Explore side effects of Thrombopoietin receptor agonists in Idiopathic thrombocytopenia purpura . 3. To study effect of thrombopoietin receptor agonists and Quality life in Idiopathic thrombocytopenia purpura patients.
Immune thrombocytopenia is an autoimmune disease in which platelets are targeted by the host immune system. Platelet depletion occurs when autoantibodies targeting glycoproteins (αIIb-β3 and GPIb) found on the surface of platelets opsonize the cells, resulting in destruction by macrophages. These antibodies can also bind to megakaryocytes and prevent complete maturation, which results in lowered levels of platelet production Vitamin D (VD) or 1,25-dihydroxyvitamin D, may be a potent immunomodulator, and it may have potential therapeutic use in many autoimmune diseases In 1991, researchers found that the production of interleukin (IL)-6 and IL-2 in cell cultures were reduced by 1,25(OH)2D3 VD deficiency is very common in children with either newly diagnosed or chronic ITP form.Correlation between hypovitaminosis D and a higher incidence of infections and autoimmune diseases was reported as well Case-Control interventional study (over time each participant will be randomized to be enrolled in one of either study arms in a random sequence). To evaluate the efficacy of VD supplementation as an adjuvant therapy in chronic ITP children and its effect on platelet count and bleeding score. The participants will be - Children from 1 year to 18 years diagnosed with Chronic ITP with platelet count <100,000/microL. Patients groups will be: Patients of Chronic ITP aged from 1year till 18 years old . in this study will be divided into 2 groups ● Group A : All patients with VIT D insufficiency(The preferred level for 25(OH)D is now recommended by many experts to be >30 ng/ml) will receive supplementary Vitamin D3(NIVAGEN Vitamin D3 Cholecalciferol 50000 IU) oral once weekly for 3 months as adjuvant therapy in combination with their treatment for ITP. ● Group B : patients in this group have sufficient VIT D level and will be divided into 2 groups : - Group 1 B :will receive their standard treatment for ITP with VIT D. - Group 2 B :will receive their standard treatment for ITP without VIT D. Complete blood picture will be made at the beginning of study then monthly to evaluate the number of platelets that expected to increase after VD supplementation
Henoch Schönlein purpura (HSP) is the most common type of vasculitis in children, with an incidence of ~10/100,000, whereas >90% of the patients develop symptoms at <10 years of age. Although HSP is generally a self-limiting disease, it may also lead to severe complications, such as intestinal intussusception, infarction and perforation, as well as end-stage renal disease. The management of HSP includes symptomatic treatment and immunosuppressive therapy in certain patients. Previous retrospective studies have reported that most patients with gastrointestinal (GI) symptoms may benefit from early usage of glucocorticoid, whereas there are still a part of HSP patients with GI did not achieved remission after administering of steroid. Therefore, the aim of the present study was to investigate the clinical features of refractory GI HSP and the clinical outcome of mycophenolate mofetil in these patients.
21 hematology centers in Turkey will participate in this noninterventional observational study. The recruitment target is 960 cases. Patients will be followed-up at least 1 year or to the end of the study according to the physicians' discretion, or withdrawal of consent. Recruitment period will be 36 months. Data will be collected via electronic case report form. Since the study cohort does not include only newly diagnosed patients, all aTTP patients will be included to the study, previous treatments and diagnosis information data could also be collected from hospital records. Patients recruitment duration: 3 years (36 months) Patients follow-up duration: 1 year (12 months)
This study conducted a prospective, multicenter, one-arm clinical trial on the combination of leflunomide and steroid therapy for refractory skin Henoch-Schonlein Purpura in children on the basis of ethical principles. A one-year follow-up was conducted to evaluate the changes in the main indicators (frequency of rash recurrence) and secondary indicators (proportion of kidney damage, proportion of joint involvement, T lymphocyte subpopulations, and inflammatory factors) before and after treatment, Exploring the safety and effectiveness of leflunomide in the treatment of refractory skin type HSP in children, it is expected that leflunomide combined with conventional treatment can improve the remission rate of HSP children's skin purpura and reduce HSP recurrence. The research results are expected to bring new treatment methods and strategies for this group of patients.